CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context
Summary: Early phase dose-finding (EPDF) trials are key in the development of novel therapies, with their findings directly informing subsequent clinical development phases and providing valuable insights for reverse translation. Comprehensive and transparent reporting of these studies is critical f...
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2025-01-01
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| author | Jan Rekowski Christina Guo Olga Solovyeva Munyaradzi Dimairo Mahtab Rouhifard Dhrusti Patel Emily Alger Deborah Ashby Jordan Berlin Oliver Boix Melanie Calvert An-Wen Chan Courtney H. Coschi Johann de Bono Thomas R. Jeffry Evans Elizabeth Garrett–Mayer Robert M. Golub Kathryn S. Hayward Sally Hopewell John D. Isaacs S. Percy Ivy Thomas Jaki Olga Kholmanskikh Andrew Kightley Shing Lee Rong Liu Israel Maia Adrian Mander Lynley V. Marshall James Matcham Richard Peck Khadija Rerhou Rantell Dawn P. Richards Lesley Seymour Yoshiya Tanaka Moreno Ursino Christopher J. Weir Christina Yap |
| author_facet | Jan Rekowski Christina Guo Olga Solovyeva Munyaradzi Dimairo Mahtab Rouhifard Dhrusti Patel Emily Alger Deborah Ashby Jordan Berlin Oliver Boix Melanie Calvert An-Wen Chan Courtney H. Coschi Johann de Bono Thomas R. Jeffry Evans Elizabeth Garrett–Mayer Robert M. Golub Kathryn S. Hayward Sally Hopewell John D. Isaacs S. Percy Ivy Thomas Jaki Olga Kholmanskikh Andrew Kightley Shing Lee Rong Liu Israel Maia Adrian Mander Lynley V. Marshall James Matcham Richard Peck Khadija Rerhou Rantell Dawn P. Richards Lesley Seymour Yoshiya Tanaka Moreno Ursino Christopher J. Weir Christina Yap |
| author_sort | Jan Rekowski |
| collection | DOAJ |
| description | Summary: Early phase dose-finding (EPDF) trials are key in the development of novel therapies, with their findings directly informing subsequent clinical development phases and providing valuable insights for reverse translation. Comprehensive and transparent reporting of these studies is critical for their accurate and critical interpretation, which may improve and expedite therapeutic development. However, quality of reporting of design characteristics and results from EPDF trials is often variable and incomplete. The international consensus-based CONSORT-DEFINE (Consolidated Standards for Reporting Trials Dose-finding Extension) statement, an extension of the CONSORT statement for randomised trials, was developed to improve the reporting of EPDF trials. The CONSORT-DEFINE statement introduced 21 new items and modified 19 existing CONSORT items.This CONSORT-DEFINE Explanation and Elaboration (E&E) document provides important information to enhance understanding and facilitate the implementation of the CONSORT-DEFINE checklist. For each new or modified checklist item, we provide a detailed description and its rationale with supporting evidence, and present examples from EPDF trial reports published in peer-reviewed scientific journals. When reporting the results of EPDF trials, authors are encouraged to consult the CONSORT-DEFINE E&E document, together with the CONSORT and CONSORT-DEFINE statement papers, and adhere to their recommendations. Widespread adoption of the CONSORT-DEFINE statement is likely to enhance the reporting quality of EPDF trials, thus facilitating the peer review of such studies and their appraisal by researchers, regulators, ethics committee members, and funders. Funding: This work is a further extension of the CONSORT-DEFINE study, which was funded by the UK Medical Research Council (MRC)-National Institute for Health and Care Research (NIHR) Methodology Research Programme (MR/T044934/1). The Clinical Trials and Statistics Unit at The Institute of Cancer Research (ICR-CTSU) receives programmatic infrastructure funding from Cancer Research UK (C1491/A25351; CTUQQR-Dec 22/100 004), which has contributed to accelerating the advancement and successful completion of this work. |
| format | Article |
| id | doaj-art-47aab48a9be448a7942bb2cf300a80e0 |
| institution | Kabale University |
| issn | 2589-5370 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | EClinicalMedicine |
| spelling | doaj-art-47aab48a9be448a7942bb2cf300a80e02025-01-22T05:43:19ZengElsevierEClinicalMedicine2589-53702025-01-0179102987CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in contextJan Rekowski0Christina Guo1Olga Solovyeva2Munyaradzi Dimairo3Mahtab Rouhifard4Dhrusti Patel5Emily Alger6Deborah Ashby7Jordan Berlin8Oliver Boix9Melanie Calvert10An-Wen Chan11Courtney H. Coschi12Johann de Bono13Thomas R. Jeffry Evans14Elizabeth Garrett–Mayer15Robert M. Golub16Kathryn S. Hayward17Sally Hopewell18John D. Isaacs19S. Percy Ivy20Thomas Jaki21Olga Kholmanskikh22Andrew Kightley23Shing Lee24Rong Liu25Israel Maia26Adrian Mander27Lynley V. Marshall28James Matcham29Richard Peck30Khadija Rerhou Rantell31Dawn P. Richards32Lesley Seymour33Yoshiya Tanaka34Moreno Ursino35Christopher J. Weir36Christina Yap37Clinical Trials and Statistics Unit at the Institute of Cancer Research, London, UKThe Institute of Cancer Research, London, UK; Royal Marsden NHS Foundation Trust, London, UKClinical Trials and Statistics Unit at the Institute of Cancer Research, London, UKDivision of Population Health, Sheffield Centre for Health and Related Research, University of Sheffield, Sheffield, UKClinical Trials and Statistics Unit at the Institute of Cancer Research, London, UKClinical Trials and Statistics Unit at the Institute of Cancer Research, London, UKClinical Trials and Statistics Unit at the Institute of Cancer Research, London, UKSchool of Public Health, Imperial College London, St Mary's Hospital, London, UKVanderbilt-Ingram Cancer Center, Nashville, TN, USABayer, Berlin, GermanyCentre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research Applied Research Collaboration West Midlands, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research Birmingham Biomedical Research Centre, NIHR Birmingham Biomedical Research Centre, Institute of Translational Medicine, University Hospital NHS Foundation Trust, Birmingham, UKDepartment of Medicine, Women's College Research Institute, University of Toronto, Toronto, CanadaCanadian Cancer Trials Group, Kingston, ON, CanadaThe Institute of Cancer Research, London, UK; Royal Marsden NHS Foundation Trust, London, UKInstitute of Cancer Sciences, CR-UK Beatson Institute, University of Glasgow, Glasgow, UKCenter for Research and Analytics, American Society of Clinical Oncology, Alexandria, VA, USADepartment of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USADepartments of Physiotherapy and Medicine, University of Melbourne, VIC, Australia; Florey Institute of Neuroscience and Mental Health, University of Melbourne, Melbourne, VIC, AustraliaOxford Clinical Research Unit, NDORMS, University of Oxford, Oxford, UKTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK; NIHR Newcastle Biomedical Research Centre, Newcastle upon Tyne Hospitals NHS Foundation Trust, Freeman Hospital, Newcastle upon Tyne, UKInvestigational Drug Branch, Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, National Institute of Health, Bethesda, MD, USAMRC Biostatistics Unit, Cambridge University, Cambridge, UK; Computational Statistics Group, University of Regensburg, Regensburg, GermanyFederal Agency for Medicines and Health Products, Brussels, BelgiumPatient and Public Involvement and Engagement (PPIE) Lead, Lichfield, UKColumbia University Mailman School of Public Health, New York, NY, USARegeneron, New York, NY, USAHCor Research Institute, Sao Paulo, BrazilCentre for Trials Research, Cardiff University, Cardiff, UKThe Institute of Cancer Research, London, UK; Royal Marsden NHS Foundation Trust, London, UKStrategic Consulting, Cytel (Australia), Perth, WA, AustraliaDepartment of Pharmacology and Therapeutics, University of Liverpool, Liverpool, UK; Hoffmann-La Roche, Basel, SwitzerlandMedicines and Healthcare Products Regulatory Agency, London, UKClinical Trials Ontario, MaRS Centre, Toronto, ON, CanadaCanadian Cancer Trials Group, Kingston, ON, CanadaFirst Department of Internal Medicine, University of Occupational and Environmental Health, Kitakyushu, JapanReCAP/F CRIN, INSERM, 5400, Nancy, France; Unit of Clinical Epidemiology, University Hospital Centre Robert Debré, Université Paris Cité, Paris, France; INSERM, Centre de Recherche des Cordeliers, Sorbonne Université, Université Paris Cité, Paris, France; HeKA Team, Centre Inria, Paris, FranceEdinburgh Clinical Trials Unit, Usher Institute, University of Edinburgh, Edinburgh, UKClinical Trials and Statistics Unit at the Institute of Cancer Research, London, UK; Corresponding author. Clinical Trials and Statistics Unit, The Institute of Cancer Research, London SM2 5NG, UK.Summary: Early phase dose-finding (EPDF) trials are key in the development of novel therapies, with their findings directly informing subsequent clinical development phases and providing valuable insights for reverse translation. Comprehensive and transparent reporting of these studies is critical for their accurate and critical interpretation, which may improve and expedite therapeutic development. However, quality of reporting of design characteristics and results from EPDF trials is often variable and incomplete. The international consensus-based CONSORT-DEFINE (Consolidated Standards for Reporting Trials Dose-finding Extension) statement, an extension of the CONSORT statement for randomised trials, was developed to improve the reporting of EPDF trials. The CONSORT-DEFINE statement introduced 21 new items and modified 19 existing CONSORT items.This CONSORT-DEFINE Explanation and Elaboration (E&E) document provides important information to enhance understanding and facilitate the implementation of the CONSORT-DEFINE checklist. For each new or modified checklist item, we provide a detailed description and its rationale with supporting evidence, and present examples from EPDF trial reports published in peer-reviewed scientific journals. When reporting the results of EPDF trials, authors are encouraged to consult the CONSORT-DEFINE E&E document, together with the CONSORT and CONSORT-DEFINE statement papers, and adhere to their recommendations. Widespread adoption of the CONSORT-DEFINE statement is likely to enhance the reporting quality of EPDF trials, thus facilitating the peer review of such studies and their appraisal by researchers, regulators, ethics committee members, and funders. Funding: This work is a further extension of the CONSORT-DEFINE study, which was funded by the UK Medical Research Council (MRC)-National Institute for Health and Care Research (NIHR) Methodology Research Programme (MR/T044934/1). The Clinical Trials and Statistics Unit at The Institute of Cancer Research (ICR-CTSU) receives programmatic infrastructure funding from Cancer Research UK (C1491/A25351; CTUQQR-Dec 22/100 004), which has contributed to accelerating the advancement and successful completion of this work.http://www.sciencedirect.com/science/article/pii/S2589537024005662Early phase trialsPhase IDose-findingDose escalation/de-escalationReporting guidanceCONSORT-DEFINE |
| spellingShingle | Jan Rekowski Christina Guo Olga Solovyeva Munyaradzi Dimairo Mahtab Rouhifard Dhrusti Patel Emily Alger Deborah Ashby Jordan Berlin Oliver Boix Melanie Calvert An-Wen Chan Courtney H. Coschi Johann de Bono Thomas R. Jeffry Evans Elizabeth Garrett–Mayer Robert M. Golub Kathryn S. Hayward Sally Hopewell John D. Isaacs S. Percy Ivy Thomas Jaki Olga Kholmanskikh Andrew Kightley Shing Lee Rong Liu Israel Maia Adrian Mander Lynley V. Marshall James Matcham Richard Peck Khadija Rerhou Rantell Dawn P. Richards Lesley Seymour Yoshiya Tanaka Moreno Ursino Christopher J. Weir Christina Yap CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context EClinicalMedicine Early phase trials Phase I Dose-finding Dose escalation/de-escalation Reporting guidance CONSORT-DEFINE |
| title | CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context |
| title_full | CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context |
| title_fullStr | CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context |
| title_full_unstemmed | CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context |
| title_short | CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trialsResearch in context |
| title_sort | consort define explanation and elaboration recommendations for enhancing reporting quality and impact of early phase dose finding clinical trialsresearch in context |
| topic | Early phase trials Phase I Dose-finding Dose escalation/de-escalation Reporting guidance CONSORT-DEFINE |
| url | http://www.sciencedirect.com/science/article/pii/S2589537024005662 |
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